The FDA has issued three draft guidances, aiming to help companies streamline development and speed approval of cell and gene therapies.
The three guidances are currently open for commentary.
Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small PopulationsThe guidance describes FDA requirements and provides considerations for the use of various clinical trial designs and endpoints to generate clinical evidence to support product licensure. The recommendations include a non-exhaustive list of trial designs that sponsors may consider for clinical trials to develop CGT products intended for use in small populations.
Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy ProductsThis guidance discusses methods and approaches for capturing post-approval real-world evidence to back safety and efficacy data for CGT products, including CGT products approved under accelerated pathways.
Expedited Programs for Regenerative Medicine Therapies for Serious ConditionsThis guidance describes the expedited programs available to sponsors of regenerative medicine therapies for serious conditions, including those products designated as RMATs. Additionally, it describes considerations in the clinical development of regenerative medicine therapies and opportunities for sponsors of such products to interact with the CBER review staff.